RT Journal Article
T1 Mesoporous Silica-Based Nanoparticles as Non-Viral Gene Delivery Platform for Treating Retinitis Pigmentosa.
A1 Valdés-Sánchez, Lourdes
A1 Borrego-González, Sara
A1 Montero-Sánchez, Adoración
A1 Massalini, Simone
A1 de la Cerda, Berta
A1 Díaz-Cuenca, Aránzazu
A1 Díaz-Corrales, Francisco J
K1 PRPF31
K1 gene therapy
K1 mesoporous silica
K1 nanoparticles
K1 retinitis pigmentosa
AB Gene therapy is a therapeutic possibility for retinitis pigmentosa (RP), in which therapeutic transgenes are currently delivered to the retina by adeno-associated viral vectors (AAVs). Although their safety and efficacy have been demonstrated in both clinical and preclinical settings, AAVs present some technical handicaps, such as limited cargo capacity and possible immunogenicity in repetitive doses. The development of alternative, non-viral delivery platforms like nanoparticles is of great interest to extend the application of gene therapy for RP. Amino-functionalized mesoporous silica-based nanoparticles (N-MSiNPs) were synthesized, physico-chemically characterized, and evaluated as gene delivery systems for human cells in vitro and for retinal cells in vivo. Transgene expression was evaluated by WB and immunofluorescence. The safety evaluation of mice subjected to subretinal injection was assessed by ophthalmological tests (electroretinogram, funduscopy, tomography, and optokinetic test). N-MSiNPs delivered transgenes to human cells in vitro and to retinal cells in vivo. No adverse effects were detected for the integrity of the retinal tissue or the visual function of treated eyes. N-MSiNPs were able to deliver a therapeutic transgene candidate for RP, PRPF31, both in vitro and in vivo. N-MSiNPs are safe for retinal delivery and thus a potential alternative to viral vectors.
SN 2077-0383
YR 2022
FD 2022-04-13
LK http://hdl.handle.net/10668/21269
UL http://hdl.handle.net/10668/21269
LA en
DS RISalud
RD Apr 7, 2025